What are the Treatment Options for Neonatal Storage Diseases?
While there is no cure for most storage diseases, several treatment options can help manage symptoms and improve quality of life:
- Enzyme Replacement Therapy (ERT): Administering the missing enzyme intravenously to reduce substrate accumulation. - Substrate Reduction Therapy (SRT): Drugs that reduce the production of the accumulated substrate. - Gene Therapy: Experimental approaches aimed at correcting the underlying genetic defect. - Supportive Care: Includes physical therapy, occupational therapy, and symptomatic treatments to manage pain, seizures, and other issues.
